[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"relapsed-or-refractory-acute-leukemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:relapsed-or-refractory-acute-leukemia":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,49],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":4},"100648501","phase-1-s243249-monotherapy-in-patients-with-relapsed-or-refractory-acute-leukemia-with-a-kmt2a-or-nup98-translocation-or-relapsed-or-refractory-aml-with-npm1c-mutation-100648501",false,"NCT07722312","S243249 Monotherapy in Patients With Relapsed or Refractory Acute Leukemia With a KMT2A or NUP98 Translocation or Relapsed or Refractory AML With NPM1c Mutation","A Phase 1\u002F2, Dose Optimization and Dose Expansion, Multicenter, Open-label Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Menin Inhibitor S243249 Monotherapy in Patients With Relapsed or Refractory Acute Leukemia With a KMT2A or NUP98 Translocation or Patients With Relapsed or Refractory Acute Myeloid Leukemia With NPM1c Mutation","Inclusion Criteria:\n\n* Aged ≥ 18 years old.\n* Negative serum pregnancy (β-hCG) test in women of childbearing potential at screening\n* Cytomorphology-confirmed diagnosis of R\u002FR acute leukemia (including AML, ALL, and mixed lineage leukemia) according to the WHO criteria in 2022. R\u002FR acute leukemia must meet at least one of the following conditions:\n* Primary refractory disease, defined as non-response to 2 courses of standard induction therapy.\n* R\u002FR disease, defined as \\> 5% blasts on bone marrow aspirate (BMA) \u002F bone marrow biopsy (BMB) after completing prior therapy.\n* Relapse after allogeneic hematopoietic stem cell transplantation (HSCT), autologous HSCT, or immunotherapy such as chimeric antigen receptor T cell therapy (CAR-T) and T cell engager (TCE).\n* Participants with secondary AML or AML transformed from myelodysplastic syndrome (MDS), myeloproliferative neoplasm (MPN), etc., can be included in the study, if they meet the above criteria after the disease has transformed into AML.\n* Confirmation of KMT2At, NUP98t, or NPM1c mutation using next generation sequencing (NGS), fluorescence in situ hybridization (FISH), or polymerase chain reaction (PCR) based test in an accredited local or central lab within 28 days before start of treatment.\n* Peripheral blood white blood cell (WBC) count ≤ 25 mm3 (hydroxyurea, steroids, or vincristine to reduce peripheral WBC count is permitted).\n* Participants will be at least 2 weeks from prior therapy (except hydroxyurea, vincristine, or steroids and prespecified prephase therapy) and recovered from nadir to no worse than Grade 1 nonhematological toxicity from the prior treatment.\n* Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.\n* Adequate electrolytes, liver, kidney, and cardiac function\n* Sexually active male or female participants of childbearing potential must agree to use 2 medically accepted forms of effective contraception, e.g., oral, parenteral, or implanted contraceptives; intrauterine devices; and barrier methods with spermicides, during the study and for 3 or 6 months after the final administration of investigational medicinal product (IMP), in males and females respectively. Egg donation is not allowed during the study or within 6 months of the last dose of S243249.\n* Male participants with women of childbearing potential (WOCBP) partners must use a condom during the study and for at least 3 months after the final administration of IMP.\n\nExclusion Criteria:\n\n* Active central nervous system (CNS) leukemia (including imaging abnormalities and cerebrospinal fluid (CSF) smear or flow cytometry indicating leukemia cells)).\n* Active disseminated intravascular coagulation (DIC).\n* Active uncontrolled infection (prophylaxis because of absolute neutrophil count \\[ANC\\] is excepted).\n* Diagnosis of acute promyelocytic leukemia (APL, M3).\n* Corrected QT interval calculated by Fridericia (QTcF) \\> 450 msec on screening ECG.\n* Participants with an increased pro-arrhythmic risk such as those with congenital long QT syndrome.\n* Uncontrolled or severe cardiovascular disease, , within 12 months.\n* Uncontrolled serious arrhythmias.\n* Clinically significant pericardial disease.\n* History of other malignancy within the past 5 years.\n* Participants who receive autologous hematopoietic stem cell transplantation (ASCT) or CAR-T therapy within 60 days of the first dose of S243249 or have not yet recovered from toxicity related to ASCT or CAR-T therapy.\n* Participants who receive allogeneic HSCT within 100 days of the first dose of S243249, still have active acute or chronic graft versus host disease (GVHD), or still require immune-modulating therapy.\n* Have an active infection of hepatitis B or hepatitis C.\n* Have advanced liver disease or cirrhosis.\n* Uncontrolled human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome related illness.\n* Pregnant and\u002For breast-feeding (lactating) women.\n* Participant has received anti-leukemia treatment, including chemotherapy, radiation therapy, targeted small molecule agents, biologic agents, immunotherapy, or any other investigational therapy (excluding hydroxyurea, or vincristine for cytoreduction) within 2 weeks prior to the first dose of S243249.\n* Previous treatment targeting menin, dose optimization phase only.\n* Any concomitant participation in another therapeutic clinical trial is prohibited. Any participation in another nontherapeutic clinical trial could be approved by the medical monitor.\n* Participants taking medications known to prolong the QT\u002FQTc interval (with the exception of necessary azole antifungals).\n* Ongoing toxicity from prior anti-leukemia therapy that has not resolved to Grade 1 7 days prior to the first dose of S243249 has to be approved by the medical monitor.\n* Uncontrolled active infection\n* Known allergy or hypersensitivity to menin inhibitors or any component of S243249.","ALL","18 Years",{"count":19,"type":20},80,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","The objective of this study is to establish the efficacy, safety, and tolerability of S243249 and to establish the recommended phase 2 dose (RP2D) of S243249 monotherapy in participants with relapsed\u002Frefractory (R\u002FR) acute leukemia with select mutations. Phase 1 dose optimization will determine the RP2D to be used in Phase 2 dose expansion. The study will include a screening period, a treatment period consisting of continuous 28-day cycles of treatment, a safety follow-up period and a long-term follow-up period. Participants may undergo blood tests, electrocardiogram (ECG), bone marrow aspirations, vital sign checks, questionnaires, and physical exams.",[27,28],"Relapsed or Refractory Acute Leukemia","Relapsed or Refractory Acute Myeloid Leukemia",[30,31,32,33,16,34,35,36],"NPM1c","KMT2At","NUP98t","AML","Acute Leukemia","Relapsed Refractory","S243249","NOT_YET_RECRUITING","2026-07-22",{"date":40,"type":41},"2026-07-23","ACTUAL",{"date":43,"type":20},"2026-09-20",{"date":45,"type":20},"2030-08-25",{"name":47,"class":48},"Servier","INDUSTRY",{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":55,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":16,"minAge":57,"maxAge":58,"enrollmentInfo":59,"targetDuration":4,"studyType":21,"phases":61,"briefSummary":63,"conditions":64,"keywords":67,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":76,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":85},"100646139","early-phase-1-clinical-study-on-the-safety-and-efficacy-of-pid23-injection-in-patients-with-relapsedrefractory-acute-leukemia-100646139","NCT07696481","Clinical Study on the Safety and Efficacy of PID23 Injection in Patients With Relapsed\u002FRefractory Acute Leukemia","A Single-center, Single-arm, Open-label Clinical Study on the Safety and Efficacy of PID23 Injection in Treating Patients With Relapsed\u002FRefractory Acute Leukemia","R\u002FR AL","Inclusion Criteria:\n\n1. Patient or legal guardian voluntarily signs the informed consent form (ICF), demonstrating understanding of the study purpose and procedures, and willingness to participate.\n2. Age 3 to 75 years, inclusive, male or female.\n3. Diagnosis of relapsed or refractory acute leukemia per guideline criteria:\n\nRelapsed: reappearance of leukemic cells in peripheral blood or bone marrow blasts ≥5% after achieving complete remission (CR); Refractory: failure to achieve CR after 2 courses of standard induction chemotherapy; relapse within 12 months after consolidation\u002Fintensification therapy; relapse after 12 months with no response to conventional chemotherapy; 2 or more relapses; extramedullary leukemia relapse or persistence; relapse after allogeneic hematopoietic stem cell transplantation.\n\n4.Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2. 5.Life expectancy ≥12 weeks. 6.Bone marrow morphology showing ≥5% primitive\u002Fimmature lymphocytes (blasts). 7.Tumor cells positive for CD19, BCMA, or CD70 expression by flow cytometry. 8.Adequate major organ function, defined as:\n\n1. Cardiac: left ventricular ejection fraction (LVEF) ≥40% by echocardiogram;\n2. Renal: serum creatinine ≤2.0× upper limit of normal (ULN), or creatinine clearance ≥50 mL\u002Fmin (Cockcroft-Gault formula);\n3. Hepatic: ALT and AST ≤3.0×ULN (≤5.0×ULN if with liver involvement); total bilirubin ≤2.0×ULN (≤3.0×ULN for Gilbert's syndrome);\n4. Pulmonary: oxygen saturation ≥92% on room air;\n5. Hematologic: absolute neutrophil count (ANC) ≥1.0×10⁹\u002FL, platelets ≥50×10⁹\u002FL, hemoglobin ≥80 g\u002FL (with bone marrow involvement, ANC ≥0.5×10⁹\u002FL, platelets ≥20×10⁹\u002FL permitted) - assessments allowed after transfusion or hematopoietic growth factor support.\n\n9\\. For women of childbearing potential, negative serum pregnancy test; all participants agree to use reliable (non-rhythm) contraceptive methods from ICF signing through 1 year post-PID23 infusion.\n\nExclusion Criteria:\n\n1. Prior treatment with CAR-T or other genetically modified cell therapies, unless the investigator determines that safety risks have been adequately excluded.\n2. Received the following anti-tumor therapies prior to PID23 infusion: Chemotherapy or molecular targeted therapy within 14 days or 5 half-lives (whichever is longer) (excluding conditioning chemotherapy and intrathecal chemotherapy; intrathecal therapy must be stopped ≥1 week prior to PID23 infusion); Radiotherapy to non-hematopoietic sites within 7 days; Radiotherapy to hematopoietic sites within 14 days.\n3. Any of the following cardiac conditions:\n\n(1) New York Heart Association (NYHA) Class III or IV congestive heart failure; (2) Myocardial infarction or coronary artery bypass grafting (CABG) within 6 months prior to enrollment; (3) Clinically significant ventricular arrhythmia, or unexplained syncope (excluding vasovagal or dehydration-related); (3) History of severe non-ischemic cardiomyopathy. 4.Active or uncontrolled infection requiring systemic therapy within 1 week prior to screening.\n\n5.Grade 2-4 acute graft-versus-host disease (GVHD) or moderate-to-severe chronic GVHD within 4 weeks prior to screening.\n\n6.Cerebrovascular accident or seizure within 6 months prior to screening. 7.Deep vein or arterial thrombosis event within 6 months prior to screening. 8.Active malignancy other than acute leukemia (excluding: inactive disease with treatment completed \\>2 years; adequately treated cervical carcinoma in situ, basal\u002Fsquamous cell skin carcinoma, localized prostate cancer post-curative surgery, ductal carcinoma in situ post-curative surgery).\n\n9.Received (attenuated) live vaccine within 4 weeks prior to screening. 10.Any other condition that, in the investigator's judgment, makes the patient unsuitable for participation in this study.","3 Years","75 Years",{"count":60,"type":20},18,[62],"EARLY_PHASE1","Relapsed or refractory acute leukemia (R\u002FR AL) is a life-threatening blood cancer with poor outcomes and limited treatment options. PID23 Injection is an innovative in vivo CAR-T therapy that delivers a viral vector encoding three targets (CD19, BCMA, and CD70) directly into patients, enabling their own T cells to generate functional CAR-T cells against leukemia cells. This single-center, single-arm, open-label, dose-escalation study (3 dose levels: 0.8×10⁹, 2×10⁹, and 4×10⁹ TU) plans to enroll 3-18 patients with R\u002FR AL aged 3-75 years, ECOG 0-2, and positive for at least one target. The primary objective is to evaluate safety, tolerability, and determine the recommended dose. Secondary objectives include preliminary efficacy (remission, survival), pharmacokinetics (CAR-T expansion), pharmacodynamics (cytokine changes), and exploratory viral clearance. After a single intravenous infusion, patients are hospitalized for ≥3 weeks, followed by monthly visits for 3 months, then every 3 months for up to 2 years. Enrollment is from May 2026 to May 2027, with follow-up through May 2029. The study is conducted at Zhujiang Hospital of Southern Medical University (PI: Prof. Li Yuhua).",[27,65,66],"Relapsed or Refractory Acute Lymphoblastic Leukemia","Relapsed or Refractory Acute Myeloid Leukemia (AML)",[68,69,34,70,71,33,72,73,74],"PID23","In vivo CAR-T","Relapsed","Refractory","B-ALL","Acute Myeloid Leukemia","Acute lymphoblastic leukemia","2026-07-09",{"date":77,"type":41},"2026-07-10",{"date":79,"type":20},"2026-08-01",{"date":81,"type":20},"2030-03-31",{"name":83,"class":84},"Zhujiang Hospital","OTHER",1]